Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.
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Summary
A gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gammac Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells, which provided full correction of disease phenotype and clinical benefit.
- Type
- article
- Published
- 2000-04-28
- Cited by
- 2,674
- References
- 27
- OpenAlex
- https://openalex.org/W10784449
- Semantic Scholar
- https://api.semanticscholar.org/CorpusID:29935652
Keywords
Computer science
References
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- gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function.
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- Atypical X-linked severe combined immunodeficiency due to possible spontaneous reversion of the genetic defect in T cells.
- The sizes of the CDR3 hypervariable regions of the murine T-cell receptor beta chains vary as a function of the recombined germ-line segments.
- T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates
- Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA− Immunodeficient Patients
- Natural and engineered disorders of lymphocyte development.
- A new chance for SCID gene therapy
- Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells
- Engraftment of gene–modified umbilical cord blood cells in neonates with adenosine deaminase deficiency
- The interleukin-2 receptor gamma chain: its role in the multiple cytokine receptor complexes and T cell development in XSCID.
- Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells.
- Retroviral marking of canine bone marrow: long-term, high-level expression of human interleukin-2 receptor common gamma chain in canine lymphocytes.
- Retroviral-mediated gene correction for X-linked severe combined immunodeficiency.
- Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency.
- Correction of interleukin-2 receptor function in X-SCID lymphoblastoid cells by retrovirally mediated transfer of the gamma-c gene.
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- 2. Promoter Trapping Reveals Significant Differences in Integration Site Selection Between MLV and HIV Vectors
- Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.
- Lentiviral vectors for gene therapy of HIV-induced disease.
- Correction of phenotype in a thalassemia mouse model using a nonmyeloablative marrow transplantation regimen.
- La thérapie génique: sa place actuelle et son avenir
- Treatment options for genetically determined immunodeficiency.
- Recurrent infections in a nine-month-old male infant.
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- The development of gene therapy: from monogenic recessive disorders to complex diseases such as cancer.
- Murine retroviral but not human cellular promoters induce in vivo erythroid-specific deregulation that can be partially prevented by insulators.
- Hematopoietic stem cell-targeted neonatal gene therapy reverses lethally progressive osteopetrosis in oc/oc mice.
- Monitoring for potential adverse effects of prenatal gene therapy: genotoxicity analysis in vitro and on small animal models ex vivo and in vivo.
- Arthritis gene therapy's first death
- Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
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